Drug intelligence / Profile preview

gad65 aav vector

Development stage
Preclinical
Lead developer
MeiraGTx
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies, Recombinant Proteins and Enzymes
Administration
Intrathecal, Epidural, Direct Nerve Injection (e.g., Sciatic Nerve Or Dorsal Root Ganglion)
01

Overview

gad65 aav vector is a recombinant adeno-associated virus (AAV) vector engineered to express the human glutamate decarboxylase 65 (GAD65) enzyme in target tissues, particularly neurons of the dorsal root ganglion (DRG). Its primary mechanism of action is mediated gene therapy: the vector delivers the GAD65 cDNA, resulting in sustained GAD65 protein expression and the subsequent increase of local gamma-aminobutyric acid (GABA) synthesis. This elevation in GABAergic transmission provides analgesic effects and neuroprotection by counteracting nociceptive neuron hyperactivity, neuroinflammation, and neurodegeneration. The gad65 aav vector is being developed chiefly as a gene therapy for neuropathic pain and is supported by multiple preclinical studies that demonstrate efficacy in animal models of chronic and diabetic neuropathic pain through long-term pain reduction and improvement of neuroinflammatory markers[1][2].

Other names
gad65 aavgad-65 aavgad 65 aavaav-gad65aav-gad-65aav-gad 65rAAV2-GAD65rAAV-2-GAD65rAAV 2-GAD65adeno-associated virus gad65 vector
02

Targets

GAD2 (Glutamic Acid Decarboxylase 65)

Beyond the preview

Go deeper on gad65 aav vector.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Full profile access

Follow clinical development from study design and recruitment through results.

  • Trial phase
  • Status
  • Readouts

Indications & development

Full profile access

Explore development by indication, patient population, and geography.

  • Indications
  • Development status
  • Countries

Licensing & deals

Full profile access

Trace asset ownership, licensing agreements, and commercial partnerships.

  • Partners
  • Deal terms
  • Milestones

Patents & exclusivity

Full profile access

Explore the patent landscape and regulatory exclusivity around an asset.

  • Patents
  • Expiration dates
  • Exclusivity

Competitive landscape

Full profile access

Compare development programs by target, modality, and indication.

  • Competing assets
  • Targets
  • Development stage

Research & analysis

Full profile access

Connect source evidence and development news to your research questions.

  • Publications
  • News
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on gad65 aav vector.

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call